FOLD - EC approves Amicus' Pombiliti therapy for late-onset Pompe disease

2023-03-27 07:47:09 ET

• Amicus Therapeutics ( NASDAQ: FOLD ) has received approval from the European Commission for its Pombiliti enzyme replacement therapy.
• The Commission approved Pombiliti as a long-term enzyme replacement therapy, in combination with miglustat, for adults with late-onset Pompe disease.
• Pompe is an inherited lysosomal disorder caused by deficiency of the enzyme acid alpha-glucosidase. Late-onset Pompe disease can be severe and debilitating, including progressive muscle weakness throughout the body, particularly the skeletal muscles and muscles controlling breathing, that worsens over time.
• The EC approval is based on clinical data obtained from a Phase 3 pivotal study and follows the positive opinion previously granted by the Committee for Medicinal Products for Human Use (CHMP).
• Amicus ( FOLD ) has also ubmitted analytical testing for miglustat, with CHMP's opinion for the same expected in the second quarter of 2023.

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